A recent article published in the Journal of the American Medical Association (JAMA) discussed a comparison of clinical trials for orphan vs. non-orphan oncology drugs. The article questioned whether safety and efficacy standards were as stringent in the orphan trials as in the non-orphan trials. A news story circulated after the article was published went [...]
The summer of 2010 saw remarkable activity related to the development of treatments for rare diseases. The U.S. Food and Drug Administration hosted a two-day public hearing for patient advocates, industry, academic researchers and others to voice their views on current procedures and possible ways to improve them.
The Senate HELP (Health, Education, Labor and [...]
The National Organization for Rare Disorders (NORD) considers today’s announcement by the Social Security Administration (SSA) of expansion of the “Compassionate Allowances” program to be extremely important to patients and families affected by rare diseases.
Since Michael Astrue became Social Security Administrator, he has provided strong leadership in addressing issues that were very difficult and discouraging for [...]
In the U.S., the health care reform process has been extremely confusing to many people. The language in the various bills is complex. And many of the proposed changes won’t take effect for several years, even if enacted now.
NORD’s position on health care reform, from the very beginning, has been that any plan needs [...]
Seriously ill patients who have exhausted all treatment options often seek access to investigational drugs by way of government-sanctioned “expanded access programs”. Recently, the U.S. Food and Drug Administration (FDA) implemented new regulations to clarify and increase patient access to investigational drugs.
However, even with the new rules, this topic is more complex than it may [...]